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Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy

The U.S. Food & Drug Administration (FDA) has cleared an investigational new drug (IND) application for the experimental gene editing therapy GPH101 by Graphite Bio to initiate a Phase 1/2 clinical trial in patients with severe sickle cell disease (SCD)

IND clearance for Phase 1/2 trial establishes Graphite Bio as the first company advancing potentially curative sickle cell gene editing therapy to restore normal haemoglobin expression by totally replacing the HbS gene with a HbA gene, in contrast to other therapies under development that simply increase fetal haemoglobin (Haemoglobin F or HbF).

GPH101 is Graphite Bio’s lead investigational therapy that will be evaluated as a potentially curative therapy for patients suffering from SCD. The investigational therapy harnesses the power of CRISPR and DNA’s natural homology-directed repair mechanisms to cut out the mutation in the sickle globin gene and paste in the correct natural (wild-type) DNA sequence, with the aim of curing SCD through directly correcting the underlying disease-causing mutation and leading to the production of completely normal red blood cells.

 »The FDA clearance to advance our first investigational therapy, GPH101, into clinical development is a tremendous milestone enabling us to rapidly advance our targeted DNA integration approach into the clinic and bringing Graphite Bio one step closer to making a difference for patients,” said Josh Lehrer, M.Phil., M.D., FACC, Chief Executive Officer at Graphite Bio. “We are eager to initiate enrollment for the CEDAR clinical trial in early 2021, which will be a historic milestone as the first experimental treatment designed to correct the mutation that is the underlying cause of sickle cell disease. With targeted gene integration, GPH101 seeks to restore normal hemoglobin expression, which has been the ultimate goal of sickle cell disease treatment for more than 70 years. »

CEDAR, a Phase 1/2, multi-center, open-label clinical study is designed to evaluate the safety, preliminary efficacy and pharmacodynamics of GPH101 in adult and adolescent patients with severe SCD. An inherited blood disorder affecting millions of people worldwide, SCD is caused by a single mutation in the ß-globin gene that leads to damaged and misshapen red blood cells resulting in anemia, blood flow blockages, intense pain, increased risk of stroke and organ damage, and reduced life span. Patient recruitment and enrollment is expected to start soon.

Source: Business Wire / Graphite Bio


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